Baseline Patient-reported Outcomes Data Research Articles

Abstract PO1-11-06: Evaluating Symptom Clusters in Patients with Breast Cancer Experiencing Aromatase Inhibitor-Associated Musculoskeletal Symptoms

Abstract Background: Although treatment with an aromatase inhibitor (AI) for 5-10 years decreases 10-year breast cancer mortality by about 40%, up to 25% of patients will stop treatment early because of treatment-related, intolerable joint and muscular pain and stiffness, called aromatase inhibitor-associated musculoskeletal symptoms (AIMSS). We previously identified that nociplastic pain, or chronic pain in the absence of tissue damage or inflammation, is associated with increased likelihood of developing AIMSS. Other studies have found that symptom clusters, such as the SPPADE symptoms (sleep disturbance, pain, physical function impairment, anxiety, depression, and low energy/fatigue) are prevalent and co-occur in patients with cancer. Here we examine the prevalence of SPPADE symptoms for patients experiencing AIMSS and willing to enroll on a clinical trial examining use of cannabidiol (CBD) to treat their symptoms. Methods: Patients with stage 0-3 hormone receptor-positive breast cancer experiencing AIMSS (defined as worst joint pain ≥4/10 during prior 7 days that developed or worsened since starting AI therapy) were eligible for enrollment in this single arm, single center phase 2 open label clinical trial (NCT04754399). Enrolled patients completed validated questionnaires at baseline and then received CBD (Epidiolex) for 15 weeks. Patient-reported outcomes (PRO) data was collected serially, including the following questionnaires which addressed SPPADE symptoms: Brief Pain Inventory (BPI) to assess pain, PROMIS Profile 29+2 v2.1 to assess multiple symptoms, and Fibromyalgia Survey (FMS) to assess nociplastic pain. Results: 39 patients enrolled on this clinical trial. The median age at enrollment was 57 (range 37-59) and 21% of patients were over age 70. The majority of patients were white (92%) and average BMI was 31 (SD 6.6). Anastrozole was the most used aromatase inhibitor (74%), followed by exemestane (18%), and letrozole (8%). 5 patients (13%) required concomitant GnRHa therapy. Baseline PRO data was obtained from 37 patients. BPI average pain score over the week prior to enrollment was 5.1 (SD 1.5) and worst pain score was 7.2 (SD 1.3). Pain interference reported on the BPI was 4.1 (SD 2.1). Compared to the general population, enrolled patients reported better physical function (T-score 60.2, SE 3.7), less depression (T-score 45.5, SE 4.7) and fatigue (T-score 46.7, SE 5.1), and similar levels of anxiety (T-score 51, SE 7.1) and sleep disturbance (T-score 49.5, SE 5.9) on the PROMIS Profile. On the FMS, 62% of patients had evidence of nociplastic pain, with average score 13.3 (SD 5.7). The median number of sites of pain in the body reported by patients was 8 (range 1-16). Conclusions: While patients enrolling on an AIMSS clinical trial reported a high degree of daily pain that regularly interferes with their activities, for most patients these symptoms did not appear to have a negative impact on mood, affect, or other symptoms that typically cluster with pain. Though it is difficult to extrapolate results from a small population, the unexpected absence of co-occurring symptoms in this population raises additional questions about the interplay of AIMSS and other symptoms commonly reported in interventional AIMSS studies. Follow-up PRO data will be obtained in this clinical trial, allowing us to examine the impact of CBD use on pain, pain interference, and other patient-reported symptoms, as well as to explore which patients are more likely to obtain benefit from CBD treatment. Funding support provided by Conquer Cancer, the ASCO Foundation and the Rising Tide Foundation for Clinical Cancer Research. Citation Format: Fleege Nicole, N. Lynn Henry. Evaluating Symptom Clusters in Patients with Breast Cancer Experiencing Aromatase Inhibitor-Associated Musculoskeletal Symptoms [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO1-11-06.

The Johns Hopkins Hope at Hopkins Clinic: Supporting the comprehensive needs of individuals with metastatic breast cancer.

452 Background: Individuals with metastatic breast cancer (MBC) may live with their disease for many years. We initiated the Johns Hopkins Hope at Hopkins Clinic, a multi-pronged quality improvement initiative that aims to optimize care for individuals with MBC. Methods: Individuals with MBC were offered participation in the Clinic, in addition to their usual care. Participants completed baseline patient reported outcome (PRO) surveys through REDCap. They were offered a menu of services, including cancer rehabilitation, supportive oncology, social work, nutrition, navigation, and access to optional services based on baseline PRO data and preferences. A medical oncologist assembled and delivered a comprehensive Care Plan synthesizing recommendations from all consultants with a focus on optimizing treatment, managing symptoms, and promoting wellness. Patients were asked to complete an evaluation survey 3 days after the clinic session, and follow-up PRO surveys at 3 and 6 months after the Care Plan was delivered. We report summary statistics of baseline demographics, tumor characteristics, and PRO scores at the pre-clinic visit. Results: From 11/2020 to 6/2022, 53 patients were referred, and 44 (83%) completed baseline surveys and participated in the Hope at Hopkins Clinic. Median age (N=44) was 58 (32-86); 32 (73%) were white, and 6 (14%) were Black; 24 (55%) had BMI>25. Tumors were mostly estrogen receptor-positive (85%) and HER2-negative (68%). Physical and mental health was not good for ≥14 days for 9/44 (20%) and 4/44 (9%), respectively. PROMIS measure scores were >1 standard deviation below average for 14/44 (32%) for Physical Health, 7/44 (16%) for Mental Health, and 10/44 (23%) for Physical Function. PHQ8 scores indicated depression for 10/44 (23%); GAD7 scores indicated anxiety for 3/44 (7%). 37 (84%) participants received an individualized Care Plan and educational materials based on identified symptoms, concerns, and interests. We will report the number and types recommendations by domain. Follow-up PRO data are available for 10 and 4 patients at 3- and 6-months, respectively. Conclusions: We have provided individualized assessment, education, and recommendations and created a comprehensive Care Plan to guide patients living with MBC. We have used the experience to implement a new multidisciplinary model, and to extend support services to patients living with MBC and their care teams.

Long-Term Outcomes Following Nonoperative Treatment of Prearthritic or Extra-Articular Hip Pain in Women.

Introduction:There is an abundance of literature focusing on morphological and surgical outcomes in women with arthritic and prearthritic hip pain. However, no studies have evaluated conservative treatment outcomes, such as physical therapy (PT) and injections, in women with prearthritic or extra-articular hip pain. The purpose of this study is to assess changes in long-term patient-reported outcome measures after nonoperative treatments in women with prearthritic or extra-articular hip pain.Methods:Twenty-nine female patients (35–65 years old) who presented to a single provider between December 1, 2012 and September 1, 2017 for prearthritic or extra-articular hip pain (Tonnis 1 or less) and had baseline patient-reported outcome data (modified Harris Hip Score [mHHS], Hip Outcome Score [HOS] activities of daily living [ADL] and sport scores, International Hip Outcome Tool-33 [iHOT-33]) available from the institutional hip registry were included. Patients underwent nonoperative treatments for intra-articular or extra-articular hip pain. A follow-up questionnaire was prospectively administered at 3–5 years after the baseline visit.Results:Most patients underwent targeted PT (n = 27; 93%) to treat intra-articular or extra-articular hip pain. Targeted PT can be defined as primarily exercise-based therapy focusing on hip and lumbar stability. Twelve patients (41%) received injections; of these, 11 were also treated with PT. Overall, significant improvements in mHHS, HOS-ADL, and iHOT-33 scores were observed (p = 0.006, 0.022, and <0.001, respectively). HOS-ADL and iHOT-33 scores improved by a median of 10.3 and 18.0 points, respectively, and were clinically significant. HOS-sport scores also improved but were not statistically significant. There were no differences in patient-reported outcomes between patients who received both PT and injections versus those who received PT, injections, or other treatments.Conclusions:Nonoperative treatments for prearthritic or extra-articular hip pain in women, specifically PT and/or injections, were associated with sustained improvements in patient-reported outcomes at 3–5 years postbaseline.

Patient reported function and symptom burden in breast cancer patients with CNS metastases.

e18635 Background: Central nervous system (CNS) metastases are associated with decreased quality of life for patients with metastatic breast cancer (MBC). Collecting patient-reported outcome (PRO) data can identify patient needs and inform patient-focused interventions. IMPACT the Brain is a care coordination program that aims to improve access to multidisciplinary care for patients with MBC and CNS metastases. The purpose of this study is to report baseline PRO data obtained as part of this program. Methods: Patients with MBC and CNS metastases are referred for enrollment in the program. PROs and caregiver assessments are collected on a voluntary basis at baseline and every 3 months. We performed a retrospective review of the baseline PRO data to determine: (1) type and severity of cancer-related symptoms reported by patients and (2) degree of burden reported by caregivers. The PROMIS Cancer Function Brief 3D Profile measures physical function (PF), fatigue (Fat), and social participation (Soc) in patients with cancer; 50.0 is the population mean and higher scores indicate a greater degree of a trait. The MD Anderson Symptom Inventory Brain Tumor (MDASI-BT) measures severity of cancer-related symptoms including interference with daily life. A score &gt; 4 represents moderate symptoms and &gt; 6 severe symptoms. The mean of the interference items represents overall symptom distress. The Short Form Zarit Burden Interview (ZBI-12) evaluates caregiver burden; a score ≥ 10 suggests mild burden while &gt; 20 suggests high burden. PRO scores were summarized using descriptive statistics. Results: Sixty patients were referred and 54 (90%) enrolled; median age was 53 years (range 31-81). 47 questionnaires were collected at baseline from 32 participants. Average PROMIS T-scores were 37.0 for PF (31.9-51.2), 59.9 for Fat (48.9-66.8), and 42.3 for Soc (32.8-57.1). Participants reported greatest difficulty with performing heavy housework and completing usual work. The mean symptom severity score reported on the MDASI-BT (n = 20) was 2.82 (SD 1.56). Fatigue was the most severe symptom (mean 5.2, SD 2.7) and 25% of patients reported moderate symptoms. The mean interference score was 3.9 (SD 2.66), with 20% of patients reporting moderate and 30% reporting severe overall symptom distress. 65% of caregivers (n = 17) had at least mild caregiver burden on the ZBI-12. Of those, almost half (45.5%) had a score consistent with high burden. Conclusions: Collecting baseline PROs was feasible and provided valuable information about the patient experience. Symptoms experienced by patients with MBC and CNS metastases, particularly fatigue, interfere with ability to carry out usual activities. The distress caused by these symptoms may not correlate with the severity reported by the patient. Given the prevalence of burden experienced by caregivers, further work is needed to explore relationships among symptom distress, symptom severity, and caregiver burden.

Baseline patient reported outcomes data shows high prevalence of overactive bladder, sexual dysfunction, depression and anxiety in Canadian men with newly diagnosed localized prostate cancer.

BackgroundResearch on prostate cancer survivorship patients has largely been on oncological outcome, incontinence and erectile dysfunction, with less data on the relationship between prostate cancer, bladder function and mental health. This study aims to elucidate the prevalence of lower urinary tract symptoms (LUTS), overactive bladder (OAB), sexual dysfunction, depression and anxiety in Canadian men with newly diagnosed localised prostate cancer.MethodsThis is a single-centre prospective cross-sectional study of men with newly diagnosed localized prostate cancer recruited from June 2017 to July 2018. The patient-reported outcomes (PRO) instruments used in this study included the international prostate symptoms score (IPSS), OAB-V8, EQ-5D™, and the Expanded Prostate Cancer Index Composite short form (EPIC-26). Clinico-pathological data were extracted from medical records. The prevalence of LUTS, OAB, sexual dysfunction, depression and anxiety were determined from the PROs.ResultsA total of 83 patients were included in this study. The median age was 63. Based on IPSS scores, 55.3% of men had mild LUTS, 36.8% had moderate LUTS and 7.9% had severe LUTS. Based on OAB-V8 scores, 55.8% of men had a score of 8 or higher, suggestive of OAB. Only 55.8% of men reported erections adequate for intercourse. 23.1% of men reported to have a moderate to big problem with depression, and 28.8% of men reported to have a degree of anxiety or depression.ConclusionsOAB is a significant problem in men with newly diagnosed localized prostate cancer, with a prevalence of 55.8% based on this study. Baseline sexual dysfunction, anxiety and depression are also prevalent in this population.

Patient-Reported Outcomes (PRO) Data from Patients (Pts) with Essential Thrombocythemia (ET) Enrolled in the MOST Study

Patient-Reported Outcomes (PRO) Data from Patients (Pts) with Essential Thrombocythemia (ET) Enrolled in the MOST Study

Development of novel emoji scale to measure patient-reported outcomes in cancer patients.

174 Background: Patient-reported outcomes (PROs), including physical function and quality of life (QOL), are important outcomes in cancer patients. Emoji are a near-universal, popular form of communication, understandable by diverse populations, including those with low health literacy. We aimed to explore whether emoji could be a valid, reliable scale for assessing PROs in cancer patients. Methods: To develop the emoji scale, 5 emoji faces were selected from the Apple emoji dictionary by study authors. 20 cancer patients participated in a card-sorting exercise to rank the emoji from negative to positive; 19 out of 20 selected the same order, which exceeded Coomb’s criteria for being a valid ordinal scale. Patients were recruited from Mayo Clinic practices. Eligibility included: adult patients with a diagnosis of lymphoma, multiple myeloma, brain, pancreatic, breast, and ovarian cancer within the past 5 years; life expectancy of &gt; 6 months; and own an iPhone version ≥ 5.0. All patients were provided with an Apple Watch and downloaded the study app. Baseline PRO data was collected on the iPhone, including single-item linear analog self-assessments (LASA) and emoji scales for fatigue, physical function, emotional well-being, and overall QOL. Results: From 2/2017-8/2017, 294 eligible patients were recruited. 25% were male, and median age was 53 years (range 20-79). The median time since diagnosis was 8.4 months (range 0-60); 63% were on active therapy, 27% were in observation post-treatment, and 10% had not been treated. All patients were “extremely” confident filling out forms, 99% had used a computer in the past 12 months, and 27% had ever used a smart watch. Emoji responses were significantly associated with validated measures of PROs; the Spearman correlations between the ordinal emoji scales and LASAs were -0.83 for fatigue, 0.72 for physical well-being, 0.72 for emotional well-being, and 0.77 for overall QOL (all p &lt; 0.0001). Of the 75 patients who have completed the study thus far, 92% reported they would use emoji scales again and 89% would recommend that others use emoji scales. Conclusions: A novel ordinal emoji scale is strongly associated with accepted measures of PROs in cancer patients and should be studied further.

Independent predictors of reliability between full time employee-dependent acquisition of functional outcomes compared to non-full time employee-dependent methodologies: a prospective single institutional study.

The prospective acquisition of reliable patient-reported outcomes (PROs) measures demonstrating the effectiveness of spine surgery, or lack thereof, remains a challenge. The aims of this study are to compare the reliability of functional outcomes metrics obtained using full time employee (FTE) vs. non-FTE-dependent methodologies and to determine the independent predictors of response reliability using non FTE-dependent methodologies. One hundred and nineteen adult patients (male: 65, female: 54) undergoing one- and two-level lumbar fusions at Duke University Medical Center were enrolled in this prospective study. Enrollment criteria included available demographic, clinical and baseline functional outcomes data. All patients were administered two similar sets of baseline questionnaires-(I) phone interviews (FTE-dependent) and (II) hardcopy in clinic (patient self-survey, non-FTE-dependent). All patients had at least a two-week washout period between phone interviews and in-clinic self-surveys to minimize effect of recall. Questionnaires included Oswestry disability index (ODI) and Visual Analog Back and Leg Pain Scale (VAS-BP/LP). Reliability was assessed by the degree to which patient responses to baseline questionnaires differed between both time points. About 26.89% had a history an anxiety disorder and 28.57% reported a history of depression. At least 97.47% of patients had a High School Diploma or GED, with 49.57% attaining a 4-year college degree or post-graduate degree. 29.94% reported full-time employment and 14.28% were on disability. There was a very high correlation between baseline PRO's data captured between FTE-dependent compared to non-FTE-dependent methodologies (r=0.89). In a multivariate logistic regression model, the absence of anxiety and depression, higher levels of education (college or greater) and full-time employment, were independently associated with high response reliability using non-FTE-dependent methodologies. Our study suggests that capturing health-related quality of life data using non-FTE-dependent methodologies is highly reliable and maybe a more cost-effective alternative. Well-educated patients who are employed full-time appear to be the most reliable.

A Systematic Review of Patient-reported Outcomes in Randomized Controlled Trials of Unplanned General Surgery.

Unplanned general surgery represents a major workload and requires comprehensive evaluation with appropriate outcomes. This study aimed to summarize current reporting of patient-reported outcomes (PROs) in randomized clinical trials (RCTs) in unplanned general surgery. A systematic review identified RCTs reporting PROs in the commonest six areas of unplanned general surgery. Details of the PRO measures were examined using the CONSORT extension for PRO reporting in RCTs. Extracted information about each PRO domain included the reporting of baseline PROs, rationale for PRO selection and whether PRO findings were used in conjunction with clinical outcomes to inform treatment recommendations. The internal validity of included studies was assessed using the Cochrane risk of bias tool. 12,519 abstracts were screened and 20 RCTs containing data from 2037 patients included. Included studies used 14 separate PRO measures covering 35 different health domains. A visual analogue assessment of pain was most frequently reported (n = 13). Reporting of baseline PRO data was uncommon (11/35 PRO domains). The rationale for PRO data collection and a PRO-specific hypothesis were provided for 9 (25.7 %) and 5 (14.3 %) domains, respectively. Seventeen RCTs (85.0 %) used the PRO data alongside clinical outcomes to inform treatment recommendations. Of the 116 risk of bias assessments, 77 (66.0 %) were judged as high or unclear. There is a lack of well designed, and conducted RCTs in unplanned general surgery that include PROs. Future work to define relevant PROs and methods for optimal assessment are needed to inform health care decision-making.

Assessing Patient Reported Outcomes Measures via Phone Interviews Versus Patient Self-Survey in the Clinic: Are We Measuring the Same Thing?

Longitudinally following patients requires a full-time employee (FTE)-dependent data inflow infrastructure. There are efforts to capture patient-reported outcomes (PROs) by the use of non-FTE-dependent methodologies. In this study, we set out to assess the reliability of PRO data captured via FTE-dependent compared with non-FTE-dependent methodologies. A total of 119 adult patients (65 men) who underwent 1-and 2-level lumbar fusions at Duke University Medical Center were enrolled in this prospective study. Enrollment criteria included available demographic, clinical, and PRO data. All patients completed 2 sets of questionnaires--the first a phone interviews and the second a self-survey. There was at least a 2-week period between the phone interviews and self-survey. Questionnaires included the Oswestry Disability Index (ODI), the visual analog scale for back pain (VAS-BP), and the visual analog scale for leg pain (VAS-LP). Repeated-measures analysis of variance was used to compare the reliability of baseline PRO data captured. A total of 39.49% of patients were smokers, 21.00% had diabetes, and 11.76% had coronary artery disease; 26.89% reported history of anxiety disorder, and 28.57% reported history of depression. A total of 97.47% of patients had a high-school diploma or General Education Development, and 49.57% attained a 4-year college degree or postgraduate degree. We observed a high correlation between baseline PRO data captured between FTE-dependent versus non-FTE dependent methodologies (ODI: r = -0.89, VAS-BP: r = 0.74, VAS-LP: r = 0.70). There was no difference in PROs of baseline pain and functional disability between FTE-dependent and non-FTE-dependent methodologies: baseline ODI (FTE-dependent: 47.73 ± 16.77 [mean ± SD] vs. non-FTE-dependent: 45.81 ± 12.11, P = 0.39), VAS-LP (FTE-dependent: 6.13 ± 2.78 vs. non-FTE-dependent: 6.46 ± 2.79, P = 0.36) and VAS-BP (FTE-dependent: 6.33 ± 2.90 vs. non-FTE-dependent: 6.53 ± 2.48, P = 0.57). Our study suggests that there is great reliability between PRO data captured between FTE-dependent and non-FTE-dependent methodologies.

Investigating the collection and assessment of patient-reported outcome data amongst unplanned surgical hospital admissions: a feasibility study.

BackgroundRandomised controlled trials (RCTs) in surgery can be challenging to conduct, and trials in the emergency surgical setting when patients have unplanned hospital admissions are particularly difficult. One area of challenge is capturing baseline patient-reported outcome (PRO) data. This study examined the feasibility and optimal methods for the collection of baseline and follow-up PRO data in the setting of unplanned surgical hospital admissions.MethodsClinically stable adult patients with unplanned admissions through the day and night under the care of general surgeons at two acute NHS trusts were approached during working week days and asked to complete validated PRO measures (European Quality of Life-5 Dimension, Short Form-12, and Gastrointestinal Quality of Life Index) on admission and 6 weeks following discharge. Feasibility of PRO data collection was determined by the proportions of admitted patients eligible and recruited and by questionnaire-response rates at baseline and follow up. Reasons for non-recruitment and non-completion of questionnaires were sought and recorded.ResultsThere were 276 admissions, of whom 235 (85.1 %) were eligible. Reasons for ineligibility were the following: age under 18 years old (n = 5, 1.8 %), non-surgical presenting complaint (n = 6, 2.2 %) and clinical instability (n = 30, 10.9 %). One hundred and sixty-six patients (70.6 %) were recruited (98 female, 59.0 %); median age 53, range 19–100). Common reasons for non-recruitment included patients being discharged home before approached by researchers (n = 29, 12.3 %) or declining participation because they felt unwell (n = 15, 6.4 %). The most common reason for admission to the hospital was abdominal pain (n = 120, 72.3 % recruited patients), of whom 50 (30.1 %) required operative intervention. Baseline PRO data was obtained from 153 patients (93.3 %), and 74 (48.4 %) returned follow-up questionnaires.ConclusionsCollection of baseline PRO data amongst unplanned admissions in general surgery is feasible. Methods for optimising retention and follow up are needed.

The humanistic burden of head and neck cancer: a systematic literature review.

Head and neck cancer (HNC) and its treatment can affect communication, nutrition, and physical appearance, and the global impact of this disease on patients' quality of life may be substantial. The aim of this systematic literature review was to describe the impact of HNC and its treatment on the physical, emotional, and social well-being of patients over time, by examining longitudinal studies of patient-reported outcomes (PRO) evaluating these domains. Databases (MEDLINE and Embase) were searched to identify studies published in English between January 2004 and January 2014 analyzing the humanistic aspects of HNC in adult patients. Additional relevant publications were identified through manual searches of abstracts from recent conference proceedings. Of 1,566 studies initially identified, 130 met the inclusion criteria and were evaluated in the assessment. Investigations using a variety of PRO instruments in heterogeneous patient populations consistently reported that PRO scores decrease significantly from diagnosis through the treatment period, but generally recover to baseline in the first year post-treatment. This trend was observed for many functional domains, although some side effects, such as xerostomia, persisted well beyond 1 year. In addition, considerable evidence exists that baseline PRO scores can predict clinical endpoints such as overall and progression-free survival. Many aspects of HNC, both disease and treatment specific, profoundly affect patients' quality of life. Improved knowledge of these effects on PRO may allow for more informed treatment decisions and can help physicians to better prepare patients for changes they may experience during therapy. Furthermore, the predictive value of baseline PRO data may enable healthcare providers to identify at-risk patients in need of more intensive intervention.

The Importance of Outcome Measurement in Orthopaedics

The Importance of Outcome Measurement in Orthopaedics

Use of Patient-Reported Outcomes (PROs) to Assess Prognosis, Toxicity, and Response: The Bortezomib Experience in Relapsed and Refractory Multiple Myeloma (MM).

PROs provide patients' own appraisal of their health status and can be used to measure aspects of health that are generally not captured by ‘traditional' clinical measures such as physician assessments and test results. This study analyzed PRO data from a multicenter, pivotal phase II trial of bortezomib in 202 patients (pts) with relapsed, refractory MM (NEJM 2003; 348:2609–17). PRO questionnaires (EORTC-QLQ C30 and MY24, FACT/GOG-Neurotoxicity (Ntx) and FACIT-Fatigue subscales) were administered at the screening visit (baseline) and day 1 of cycles 3, 5, 7 and at the end of the study.Results: In this population with relapsed, refractory MM, poorer baseline pre-treatment multi-dimensional quality of life (QoL) scores were significantly correlated (range of r= 0.22 to r= 0.77) with fatigue, pain, dyspnea, appetite loss, neurotoxicity, MM disease symptoms, and treatment side effects. Clinical response to bortezomib (Complete Response or Partial Response) was associated with statistically significant (p<0.05) improvements in global QoL, physical, emotional and social functioning, fatigue, dyspnea, constipation, disease symptoms, social support and future perspective, whereas pts with a minimal response, no change or disease progression usually reported stable or worsening scores. Despite concern about peripheral neuropathy (12% for grade 3), FACT/GOG-Ntx change scores were stable in all responder groups. The prognostic value of baseline PRO data relative to clinical information was assessed using Partial Least Square (PLS) regression. First a model predicting death during the study (17 patients died during the study out of 202; 8.42%) identified that at baseline high total serum protein, bone marrow plasma cells and ß2-microglobulin and low platelet count, serum albumin, hemoglobin, Karnofsky score, body surface area and weight were predictive of death. In a separate model, the following baseline PRO scores predicted death during the study: high fatigue, appetite loss, treatment side effects, sleep disturbance, dyspnea and pain scores and low FACIT-Fatigue, physical, role, social, global QoL, cognitive, emotional and FACT/GOG-Ntx scores. The combination of the clinical and PRO PLS components into a hierarchical PLS regression resulted in an increase in the predictive value of the model, suggesting complementary information.Conclusion: PRO data can serve as independent measures of clinical benefit, toxicity, and even mortality risk, complementing traditional physician assessments and test results. In this trial of relapsed and refractory MM pts, PRO results suggested improvements in QoL for clinical responders, tolerable side effects, and independent prognostic value for mortality.